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Drugs in development · updated 30 October 2026

Topotecan: clinical trials and development status

Other names and codes: Hycamtin, Potactasol

NLM verified identityApproved in the US (FDA)Authorised in the EU (EMA) since 12 November 1996

As of 30 October 2026 Topotecan is in 74 active clinical trials (recruiting or ongoing, phases I–III) for 39 diseases. The most advanced phase is III.

74Active trials
IIIMost advanced phase
39Diseases studied

Summary updated every week · data as of 30 October 2026. In Phuturdex you can follow Topotecan and get an email at every change or safety alert.

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Diseases studied

For each disease: the drug’s most advanced phase and active trials, with the reference trial on ClinicalTrials.gov. Showing 30 of 39 diseases.

DiseaseMost advanced phaseActive trialsReference trial
Lung small cell carcinomaIII20NCT07099898
Ovarian neoplasmIII13NCT07286266
Fallopian tube neoplasmIII10NCT07213804
NeuroblastomaIII10NCT06172296
Lung neoplasmIII4NCT07099898
RetinoblastomaIII4NCT04799002
GanglioneuroblastomaIII3NCT06172296
MetastasisIII3NCT07213804
NeoplasmIII3NCT07365241
Ovarian epithelial carcinomaIII3NCT07218809
Uterine cervix neoplasmIII3NCT07797153
Peritoneal neoplasmIII1NCT07213804
Pleuropulmonary blastomaIII1NCT06647953
Acute myeloid leukemiaII2NCT03289910
Brain NeoplasmsII2NCT05535166
Endometrial neoplasmII2NCT06548763
Ewing sarcomaII2NCT07321912
MedulloblastomaII2NCT05535166
Neuroendocrine carcinomaI–II2NCT05652686
Proliferative vitreoretinopathyII2NCT06818721
Rhabdoid tumorII2NCT02114229
Atypical chronic myeloid leukemiaII1NCT03289910
Chronic myelomonocytic leukemiaII1NCT03289910
Myelodysplastic–myeloproliferative diseasesII1NCT03289910
MyelofibrosisII1NCT03289910
Non-small-cell lung carcinomaII1NCT05800587
OsteosarcomaII1NCT07321912
Polycythemia veraII1NCT03289910
Thrombocythemia, EssentialII1NCT03289910
Wilms TumorII1NCT04322318

Everything on Topotecan

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Source: Phuturdex analysis of ClinicalTrials.gov (U.S. National Library of Medicine), FDA and EMA data, updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III. Diseases are those named by each trial (NLM MeSH terms). The estimated launch, when shown, is a Phuturdex calculation described on the Method page, not official data.

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